Odevixibat Medicine 06 June 2023 Extended indication Kayfanda is indicated for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older. Therapeutic value No estimate possible yet Total cost € 855,000.00 Registration phase Registered Product Active substance Odevixibat Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Liver diseases Extended indication Kayfanda is indicated for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older. Current proprietary name Already available biosimilars / generics Proprietary name Kayfanda Manufacturer Ipsen Portfolio holder Ipsen Mechanism of action Other Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2022 Expected Registration September 2024 Orphan drug No Registration phase Registered Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 3 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Kamath B et al. Systematic Review: The Epidemiology, Natural History, and Burden of Alagille Syndrome. J Pediatr Gastroenterol Nutr 2018 (1); CBS Statline. 2022 (2); Additional remarks De prevalentie bij geboorte is 1 in 50:000 (1). Bij een gemiddeld aantal geboortes van 170.000 in Nederland worden er gemiddeld drie patiënt per jaar verwacht(2). De plek van dit geneesmiddel is waarschijnlijk bij patiënten die geen levertransplantatie hoeven te ondergaan. Als je wel een levertransplantatie moet ondergaan dan is het mogelijk een tijdelijke therapie. Expected cost per patient per year Cost € 250,000.00 - 320,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Zorginstituut Nederland. GVS-rapport odevixibat (Bylvay®). 2022; Additional remarks In de budget impact analyse van odexivibat voor de indicatie PFIC zijn twee verschillende scenario's geschetst aan de hand van de dosis die patiënten zullen ontvangen. In het minimale scenario wordt er ongeveer uitgegaan van gemiddeld €250.000 per patiënt per jaar. In het maximale scenario wordt uitgegaan van gemiddeld €320.000 per patiënt per jaar. Potential total cost per year Total cost € 855,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Andere lopende fase III studie: Biliary atresia References Adis Insight Additional remarks Other information Additional remarks